The U.S. FDA has granted Breakthrough Therapy Designation to investigational ER-004 protein replacement therapy for EspeRare’s treatment for the prenatal treatment of XLHED.
The Breakthrough Therapy Designation was granted following the promising results from three XLHED subjects who were treated by Professor Holm Schneider with a course of ER-004 intra-amniotic injections during the third trimester of pregnancy. These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants, were published in the New England Journal of Medicine.
The FDA Breakthrough Therapy Designation intends to expedite the development and review of drugs for serious or life-threatening conditions. To be granted, it requires preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least one clinically-significant endpoint compared to available therapy. This Designation allows a program to benefit from all of the FDA's fast-track program features, including proactive FDA guidance on an optimal drug development plan, organizational commitment involving FDA senior managers, and eligibility for rolling and priority reviews.
